Full Press Release Details
Alterity Therapeutics Completes Last Patient
Visit in ATH434-202 Open-Label Phase 2 Trial in Multiple System Atrophy
Disease Modifying Drug Candidate Targeting Parkinsonian Disorders -
Expected Mid-Year 2025 -
MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA
- 27 March 2025: Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) ("Alterity" or "the Company"), a biotechnology
company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that the last patient
in the ATH434-202 Phase 2 trial has completed the study. The ATH434-202 is an open label study designed to evaluate the safety, efficacy
and target engagement of ATH434 in participants with advanced multiple system atrophy (MSA).
"Following the positive results from our
randomized, double-blind Phase 2 trial1, we are pleased to announce that the last participant has now completed all clinical
evaluations in our open-label study of advanced MSA," said, David Stamler, M.D., Chief Executive Officer of Alterity. "The
202 study gives us the opportunity to evaluate the effects of ATH434 treatment in a population that faces severe challenges due to the
stage of their illness. The data from this study will help guide our development program given the differences between the 202 study and
the double-blind trial. I greatly appreciate the contributions of the trial participants and thank them for their participation. We look
forward to reporting topline data from this study in mid-year 2025."
About ATH434-202 Phase 2 Clinical Trial
The ATH434-202 Phase 2 clinical trial is an open
label study, entitled "A Biomarker Study of ATH434 in Participants with MSA." The Biomarker trial enrolled 10 individuals
with advanced MSA. ATH434-202 study participants received treatment with ATH434 at the 75 mg dose for 12-months. The study will assess
the effect of ATH434 treatment on neuroimaging and protein biomarkers to evaluate target engagement, in addition to clinical measures,
safety, and pharmacokinetics. The selected biomarkers, including brain volume, iron and aggregating -synuclein, are important contributors
to MSA pathology and are appropriate targets to demonstrate drug activity. The primary objective of this study is to evaluate the impact
of 12 months treatment with ATH434 on brain volume in a more advanced patient population than was studied in Alterity's randomized
Phase 2 trial. Additional information on the open label Phase 2 trial can be found at clinicaltrials.gov NCT05864365.
Alterity's lead candidate, ATH434, is an
oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. ATH434 has been shown preclinically
to reduce -synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain. As an iron chaperone,
it has excellent potential to treat Parkinson's disease as well as various Parkinsonian disorders such as Multiple System Atrophy
(MSA). ATH434 successfully completed Phase 1 studies demonstrating the agent is well tolerated and achieved brain levels comparable to
efficacious levels in animal models of MSA. ATH434 recently announced positive results from the randomized, double-blind, placebo-controlled
Phase 2 clinical trial in patients with early-stage MSA. A second Phase 2 open-label 2 Biomarker trial in patients with more advanced
MSA is ongoing. ATH434 has been granted Orphan Drug Designation for the treatment of MSA by the U.S. FDA and the European Commission.
About Multiple System Atrophy
Multiple System Atrophy (MSA) is a rare, neurodegenerative
disease characterized by failure of the autonomic nervous system and impaired movement. The symptoms reflect the progressive loss of function
and death of different types of nerve cells in the brain and spinal cord. It is a rapidly progressive disease and causes profound disability.
MSA is a Parkinsonian disorder characterized by a variable combination of slowed movement and/or rigidity, autonomic instability that
affects involuntary functions such as blood pressure maintenance and bladder control, and impaired balance and/or coordination that predisposes
to falls. A pathological hallmark of MSA is the accumulation of the protein -synuclein within glia, the support cells of the central
nervous system, and neuron loss in multiple brain regions. MSA affects at least 15,000 individuals in the U.S., and while some of the
symptoms of MSA can be treated with medications, currently there are no drugs that are able to slow disease progression and there is no
1Multiple System Atrophy | National Institute of Neurological
Disorders and Stroke (nih.gov)
About Alterity Therapeutics Limited
Alterity Therapeutics is a clinical stage biotechnology
company dedicated to creating an alternate future for people living with neurodegenerative diseases. The Company is initially focused
on developing disease modifying therapies in Parkinson's disease and related disorders. Alterity recently reported positive data
for its lead asset, ATH434, in a Phase 2 clinical trial in participants with Multiple System Atrophy (MSA), a rare and rapidly progressive
Parkinsonian disorder. ATH434 is also being evaluated in a Phase 2 clinical trial in advanced MSA. In addition, Alterity has a broad drug
discovery platform generating patentable chemical compounds to treat the underlying pathology of neurological diseases. The Company is
based in Melbourne, Australia, and San Francisco, California, USA. For further information please visit the Company's website at
1ATH434-201 Phase 2 trial results release
Authorisation & Additional information
This announcement was authorized by David Stamler, CEO of Alterity
Therapeutics Limited.
Investor and Media Contacts:
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